Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation

RJ Mead, N Shan, HJ Reiser, F Marshall… - Nature Reviews Drug …, 2023 - nature.com
Amyotrophic lateral sclerosis (ALS) is a devastating disease caused by degeneration of
motor neurons. As with all major neurodegenerative disorders, development of disease …

Astrocytes in human central nervous system diseases: a frontier for new therapies

A Verkhratsky, A Butt, B Li, P Illes, R Zorec… - Signal transduction and …, 2023 - nature.com
Astroglia are a broad class of neural parenchymal cells primarily dedicated to homoeostasis
and defence of the central nervous system (CNS). Astroglia contribute to the …

Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial

RH Baloh, JP Johnson, P Avalos, P Allred… - Nature medicine, 2022 - nature.com
Amyotrophic lateral sclerosis (ALS) involves progressive motor neuron loss, leading to
paralysis and death typically within 3–5 years of diagnosis. Dysfunctional astrocytes may …

ALS genetics: gains, losses, and implications for future therapies

G Kim, O Gautier, E Tassoni-Tsuchida, XR Ma… - Neuron, 2020 - cell.com
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder caused by the loss
of motor neurons from the brain and spinal cord. The ALS community has made remarkable …

Decoding ALS: from genes to mechanism

JP Taylor, RH Brown Jr, DW Cleveland - Nature, 2016 - nature.com
Amyotrophic lateral sclerosis (ALS) is a progressive and uniformly fatal neurodegenerative
disease. A plethora of genetic factors have been identified that drive the degeneration of …

Development of multifunctional molecules as potential therapeutic candidates for Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis in the …

MG Savelieff, G Nam, J Kang, HJ Lee, M Lee… - Chemical …, 2018 - ACS Publications
Neurodegenerative diseases pose a substantial socioeconomic burden on society.
Unfortunately, the aging world population and lack of effective cures foreshadow a negative …

Autophagy and ALS: mechanistic insights and therapeutic implications

JP Chua, H De Calbiac, E Kabashi, SJ Barmada - Autophagy, 2022 - Taylor & Francis
Mechanisms of protein homeostasis are crucial for overseeing the clearance of misfolded
and toxic proteins over the lifetime of an organism, thereby ensuring the health of neurons …

Inflammation in ALS/FTD pathogenesis

ME McCauley, RH Baloh - Acta neuropathologica, 2019 - Springer
Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are
neurodegenerative diseases that overlap in their clinical presentation, pathology and …

Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS

T Nagata, CA Dwyer, K Yoshida-Tanaka, K Ihara… - Nature …, 2021 - nature.com
Achieving regulation of endogenous gene expression in the central nervous system (CNS)
with antisense oligonucleotides (ASOs) administered systemically would facilitate the …

Non-neuronal cells in amyotrophic lateral sclerosis—from pathogenesis to biomarkers

BF Vahsen, E Gray, AG Thompson, O Ansorge… - Nature Reviews …, 2021 - nature.com
The prevailing motor neuron-centric view of amyotrophic lateral sclerosis (ALS)
pathogenesis could be an important factor in the failure to identify disease-modifying therapy …